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2009: Substantial Progress Propelled MS Research Forward

Posted Dec 28 2009 9:40am
Dec 15, 2009

This year saw exciting research progress, with unprecedented opportunities on the horizon and more scientists than ever working on important questions. While the economic environment resulted in an unfortunate slowing of funding, creating gaps in planned expenditures to support MS research, we hope this impact will be of short duration.

Despite these challenges, the National MS Society continues to propel research forward and move us closer to a world free of MS.

In 2009 the Society provided over $33.5 million to support 345 new and ongoing projects in our research portfolio, plus $1.5 million for Fast Forward, our drug development subsidiary which continues to attract new funding streams. In addition, thanks to the efforts of our MS activists, $5 million has been specifically set aside for funding MS research out of the 2009 Department of Defense budget.

The Society has nimbly pursued promising opportunities when they arise, such as the recent request for research applications to expeditiously examine the potential impact of the chronic cerebrospinal venous insufficiency (CCSVI) hypothesis on disease process in MS.

Here is just a small sample of the many important, potentially high-impact research results that occurred this year, which support our three research goals: stopping MS, reversing the damage and restoring function, and ending MS forever.

STOPPING MS

· Several papers published this year provided more evidence that starting on MS therapies early after disease onset, or even before a definite diagnosis, can pay off later by delaying disease progression. In addition, results of clinical trials of oral therapies and therapies with infrequent dosing regimens were presented at medical meetings and in journal articles this year. So far, some appear to have benefit against MS attacks, and safety profiles look acceptable.

· Novartis International AG announced that oral FTY720 (fingolimod) significantly reduced relapse rates and slowed disability progression over two years in a large-scale, phase 3 trial in relapsing-remitting MS. According to the company, safety data confirmed a positive benefit-risk profile, and the company plans to seek marketing approval at the end of calendar year 2009.

· An oral drug was submitted to the FDA for marketing approval in September 2009. In a large-scale clinical trial, cladribine tablets significantly reduced relapse rates and other disease activity in people with relapsing-remitting MS. If the FDA application is successful, it would be the first approved oral disease-modifying therapy for MS.

· Positive results were announced by sponsors of a clinical trial of the experimental IV monoclonal antibody ocrelizumab, which significantly reduced disease activity on MRI scans in a study of 220 people with relapsing-remitting MS. Since this was a phase 2 study, additional research will be needed to further determine this drug’s safety and benefits.

· An international task force convened by the Society published a landmark paper in the journal Multiple Sclerosis to guide neurologists through the complex process of distinguishing MS from look-alike disorders (“differential diagnosis”). This paper was the most frequently downloaded paper from the journal’s Website this year.

· Fast Forward made its first investments in promising agents. This effort to speed the delivery of new treatments to people with MS has already attracted over $20 million in new funding.

· A small NIH-supported study by Stanford University researchers found that women who breastfed their babies exclusively (without giving supplemental bottles) for at least the first two months post-partum were less likely to have an MS relapse than those who did not breastfeed or who did not breastfeed exclusively during the first two months.

· Multiple previous studies have documented that smoking cigarettes increases the risk of developing MS. Harvard researchers have now tracked hundreds of smokers, ex-smokers and never-smokers, all of whom had MS, for an average of over three years. They reportedtwo other separate studies, State University of New York, Buffalo researchers reported links between smoking and brain tissue damage observed on MRI scans of people with MS, and Karolinska Institute investigators in Sweden confirmed negative effects of smoking, but found no association between the use of Swedish snuff and the risk of developing MS. This latter study suggests that a component of cigarettes other than nicotine is responsible for the increased risk of developing MS. that disability progressed more quickly in smokers. And in several measures, ex-smokers did not differ substantially from never-smokers, suggesting that quitting may delay MS progression. In

· Early, small-scale trials are underway or getting started to test the potential of a special type of adult stem cell found in the bone marrow, called mesenchymal stem cells. Although these cells have the potential for both turning down MS immune attacks and stimulating nervous system repair, it’s still too early to know whether this approach will prove safe and beneficial.

· To help ensure that stem cell trials are conducted in a scientifically sound way that will move the field forward, the Society convened an international meeting with our counterparts in the U.K., Italy, and France to develop guidelines for stem cell trials, which are expected to be published in 2010.

· An international team funded in part by the National MS Society reversed MS in mice by administering lisinopril, a drug commonly used to lower blood pressure. A drug used to treat diabetes (metformin) also improved MS-like disease in mice in a study by Society-funded researchers from Medical University of South Carolina and Mayo Clinic. And researchers from Canada reported that mice recovered from MS-like disease when administered a compound (called “GIFT15”)formed by fusing two immune proteins.

· A European collective of imaging experts known as “MAGNIMS” published new information on imaging and clinical findings that might help explain why some people experience a mild course of MS, also known as “benign” MS. They recommended that cognitive functions, not just physical functions, be taken into account when labeling a person’s MS as benign.

· The first large-scale clinical (“phase II”) trial of the sex hormone estriol in MS, funded by the National MS Society and especially its Southern California chapter, along with the NIH, continued to recruit women with MS to participate, expanding the number of centers to 16 across the US. The two-year trial could lay the groundwork for a larger, definitive trial that could lead to a new treatment option for women with MS.

REVERSING MS DAMAGE/RESTORING FUNCTION

· In response to reports that a phenomenon called CCSVI (chronic cerebrospinal venous insufficiency, a dysfunction of brain blood flow and/or blood drainage) may contribute to nervous system damage in MS, the Society invited investigators to apply for grant funding and created an accelerated review process to explore this lead. If confirmed, this may open up new research avenues into the underlying pathology of MS and new approaches to therapy.

· The Society gathered together 70 members of the four international Promise: 2010 Nervous System Repair Teams to share progress and plan next steps to speed clinical trials of therapies to protect and reverse neurological damage. Each team reported impressive progress, with two of the teams about to launch new, small-scale clinical trials of different types of stem cells, with separate funding.

· Members of the Society’s Nervous System Repair Team led by Professor Charles ffrench-Constant (University of Edinburgh and University of Cambridge, UK) published a paper demonstrating that a complex network of proteins that interact during brain development (called the “Wnt signaling pathway”) may also play an important role in the failure of nerve-protecting myelin to repair itself in people with MS. This could provide new clues to reversing myelin damage.

· After showing benefits for temporarily improving walking speed in all types of MS in two phase 3 clinical trials, an advisory committee for the FDA recommended marketing approval for Acorda Therapeutics’ fampridine (with the proposed new name, Amaya). If approved, this oral drug would be the first approved specifically for managing MS symptoms. The National MS Society funded early stage studies in the development of this drug.

· Two groups funded by the Society reported findings on nerve tissue injury and repair that add important information needed to stop MS progression and develop nervous system repair strategies. Mayo researchers found two enzymes that may serve as markers of progressive MS and nerve fiber injury, and investigators at Mount Sinai School of Medicine (New York) reported that a different enzyme is essential for replenishing myelin-making cells that have been depleted by MS.

· An oral drug designed to treat uncontrollable laughing and/or crying (also called pseudobulbar affect), a troubling symptom experienced by some people with MS and other neurological disorders, passed another hurdle by showing positive results in a Phase III trial. According to company sources, Zenvia™ (Avanir Pharmaceuticals) significantly reduced the rate of laughing and crying episodes and appeared to be safe and well tolerated.

ENDING MS FOREVER

· The Society launched a genetics study that should identify most of the common genes that contribute to making people susceptible to developing MS. In the short run this should set us up to discover new disease pathways that can be targeted for therapy. In the longer run, this should provide a map for preventing MS.

· Researchers at the University of Buffalo, New York and Italy reported two studies that add to growing findings linking the Epstein-Barr virus (EBV) with multiple sclerosis. One study suggested a link between EBV exposure and the loss of nerve tissue, while the other explored interactions between a person’s genes and EBV.

· Two studies published in the journal Nature Genetics identified new genes and gene regions that contribute to making people susceptible to developing MS. The findings, by the International Multiple Sclerosis Genetics Consortium and the Australia/New Zealand MS Genetics Consortium, add to a growing list of gene variations linked to MS susceptibility. The National MS Society funded the formation of the International MS Genetics Consortium and studies by the Consortium.

· For the first time, researchers in the UK and Canada found evidence of a direct interaction between vitamin D and a common genetic variant, the presence of which increases the risk of developing MS. The research highlights the importance of studying the interaction of genes and the environment to search for the underlying triggers of this complex disease.

· The Society convened an international workshop focused on strategies to find the cause of MS, as well as factors that drive progression and ways to estimate MS frequency. One outcome from the workshop is the proposed funding of a two-year pilot project to plan a large study to determine risk factors that drive MS progression and predict prognosis.

· The Society also held the first-ever Don Tykeson Fellows Conference to stimulate new research ideas and strengthen the commitment of these bright young people to MS research. Supporting fellows, who are the future of MS research, is a key component of our strategic plan.

These and other leaps forward – accomplished in the midst of the worst economic downturn since the Great Depression – made 2009 a momentous year of progress toward a world free of MS.

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